On September 30, 2000, the National Heart, Lung, and Blood Institute (NHLBI) launched the Programs of Excellence in Gene Therapy (PEGTs). The major goal of this initiative is to facilitate clinical gene therapy studies by establishing comprehensive Programs of Excellence in Gene Therapy that encourage the multidisciplinary collaboration of investigators skilled in the application of gene therapy for any one or combination of cardiovascular, pulmonary, and hematologic diseases. To accomplish this goal, this initiative seeks to create an environment that rapidly translates basic science advances into clinical application by supporting preclinical and clinical studies and providing access to specialized services, such as generating vectors for clinical use, performing morphologically based studies ranging from electron microscopy to complex image analysis, procuring and processing hematopoietic stem cells, and offering to perform primate transplantation studies. In addition, a high quality training program for M.D., M.D./Ph.D. and Ph.D. scientists will be established at each Center. Both the specialized services and training program will be available to all NHLBI-supported investigators.

The purpose of the PEGT Website is to provide information about the PEGTs' basic and clinical projects and their available resources (vector production cores, cell morphology core, hematopoietic stem cell processing core and primate stem cell transplantation core). The website will also include electronic versions of the applications that are necessary for requesting any available PEGT services or training. Applications may be filed electronically so as to expedite the process.

The administrative organization of the PEGTs is a collaborative effort between the NHLBI staff and the supported investigators. To facilitate this, several committees have been established such as an Inter-Program Steering Committee, an External Scientific Panel and Internal Advisory Committees. A Data and Safety Monitoring Board has been appointed by the Director, NHLBI to provide overall monitoring of interim data and safety issues. In addition, a Data and Coordinating Center has been established to coordinate a range of functions that is common to NHLBI and all the PEGTs.
 

Currently, there are four PEGTs:

Gene Therapy for Hemophilia

Dr. Mark Kay-Stanford University

Dr. Joseph Glorioso-University of Pittsburgh

Dr. George Stamatoyannopoulos

Dr. Ronald Crystal